Sultan N, Pope J E, Clements P J
Department of Medicine, The University of Western Ontario, Canada.
Rheumatology (Oxford). 2004 Apr;43(4):472-8. doi: 10.1093/rheumatology/keh070. Epub 2003 Dec 16.
Scoring poorly on the health assessment questionnaire (HAQ) has recently been shown to be a strong predictor of morbidity and mortality in rheumatoid arthritis (RA), while a good HAQ score is predictive of a better outcome. In patients presenting with early diffuse scleroderma prognosis is variable. Our goal was to determine possible baseline predictors of future good outcomes.
We used the raw data from two randomized controlled trials (RCTs) in early diffuse scleroderma: methotrexate (Pope et al.) and D-penicillamine (Clements et al.). Subjects in the methotrexate trial were divided into the following groups: (1) those with at least 20% improvement in the primary outcome measurements [patient global assessment, physician global assessment, UCLA skin tethering score, modified Rodnan skin score (MRSS), DLCO as % predicted and HAQ disability] at 1 yr vs (2) the others. Baseline factors (including age, gender, skin scores, physician and patient global assessments, HAQ disability and pain scores, DLCO and physical parameters) were analysed to find baseline variables strongly correlated with later improvement. These variables were explored in the D-penicillamine trial to determine if (in a separate trial) they were still predictive of improved outcome at 1 and 2 yr. Adjusted models were used to find baseline predictors of good outcome. The median HAQ-DI was 1.3 (methotrexate) and 1.0 (D-penicillamine).
A baseline HAQ disability score of less than the median was predictive of at least a 20% improvement at 1 and 2 yr with odds ratios of 1.77 to 5.05, in four of the five outcome measurements (in both groups); with strongly significant P values for 3 of 5 outcomes (UCLA skin score, MRSS, patient global skin score; P<0.02) from the methotrexate study group. These three outcomes were strongly correlated with improvement (r between 0.25 and 0.35). Although data from the D-penicillamine trial were less convincing, in both trials the less than median HAQ-DI and HAQ pain scores showed a stronger association with improved outcome, more so than age, gender, skin score and baseline global assessment.
A low baseline HAQ (defined as less than the median HAQ score) is predictive of improved outcome in diffuse scleroderma at 1 and 2 yr.
最近研究表明,健康评估问卷(HAQ)得分低是类风湿关节炎(RA)发病和死亡的有力预测指标,而HAQ得分高则预示着更好的预后。早期弥漫性硬皮病患者的预后各不相同。我们的目标是确定未来良好预后的可能基线预测因素。
我们使用了两项早期弥漫性硬皮病随机对照试验(RCT)的原始数据:甲氨蝶呤试验(Pope等人)和青霉胺试验(Clements等人)。甲氨蝶呤试验的受试者分为以下几组:(1)在1年时主要结局指标[患者整体评估、医生整体评估、加州大学洛杉矶分校皮肤束缚评分、改良Rodnan皮肤评分(MRSS)、预计DLCO百分比和HAQ残疾程度]至少改善20%的患者与(2)其他患者。分析基线因素(包括年龄、性别、皮肤评分、医生和患者整体评估、HAQ残疾和疼痛评分、DLCO和身体参数),以找出与后期改善密切相关的基线变量。在青霉胺试验中对这些变量进行了探究,以确定在另一项试验中,它们在1年和2年时是否仍能预测预后改善情况。使用校正模型来找出良好预后的基线预测因素。HAQ-DI的中位数在甲氨蝶呤试验中为1.3,在青霉胺试验中为1.0。
基线HAQ残疾评分低于中位数可预测在1年和2年时至少改善20%,在五项结局指标中的四项(两组均如此)的优势比为1.77至5.05;在甲氨蝶呤研究组的五项结局指标中有三项(加州大学洛杉矶分校皮肤评分、MRSS、患者整体皮肤评分;P<0.02)具有极显著的P值。这三项结局指标与改善情况密切相关(r在0.25至0.35之间)。虽然青霉胺试验的数据说服力较弱,但在两项试验中,HAQ-DI和HAQ疼痛评分低于中位数与预后改善的关联更强,比年龄、性别、皮肤评分和基线整体评估更为显著。
低基线HAQ(定义为低于HAQ评分中位数)可预测弥漫性硬皮病在1年和2年时预后改善。